Guideline on medicines for the treatment of amyotrophic lateral sclerosis released for a six-month public consultation

The European Medicines Agency has released a draft guideline on the clinical investigation of medicines for the treatment of amyotrophic lateral sclerosis (ALS) for a six-month public consultation.

Comments should be made using the submission form and sent no later than 31 January 2014 to cnswpsecretariat@ema.europa.eu.

ALS, often referred to as Lou Gehrig's disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Most therapeutic options for ALS target the symptoms of the disease. There are currently no medicines that slow or halt the progression of the disease.

This guideline replaces the points to consider on clinical investigation of medicinal products for the treatment of ALS .

The document focuses on the design of studies for medicines that have an impact on the course of the disease (disease-modifying treatments) as well as medicines that treat the symptoms of ALS.

Guidance is provided on the choice of meaningful outcome parameters used in studies and the clinical relevance of functional tests of disability, including motor- and respiratory-function tests and their relationship to survival.